Long-acting Cell-Based Gene Therapy, Fabry Disease and Beyond with Glafabra CEO, Dr. Chris Hopkins

In this episode of BioTalk Unzipped, hosts Gregory Austin and Dr. Chad Briscoe sit down with Glafabra CEO: Dr. Chris Hopkins, geneticist, biochemist, and biotech entrepreneur, to explore the science and strategy behind next generation cell-based gene therapies for rare diseases.

With more than 25 years of experience spanning gene augmentation, rare disease biology, CRISPR licensing, and biotech formation, Dr. Hopkins shares how autologous, ex vivo engineered cell therapies may overcome key limitations of current enzyme replacement and viral gene therapies, particularly for Fabry disease.

The conversation dives deep into:

• How lentiviral gene augmentation in patient derived cells enables sustained enzyme production

• Why redosing matters and where one time AAV therapies fall short

• The scientific rationale for early intervention, including potential newborn treatment

• Differences between autologous and emerging allogeneic approaches

• Regulatory pathways for rare disease therapies and recent FDA developments

• The role of non animal models in translational research

• Montana’s early access therapy law and its broader implications

• Building biotech platforms amid a challenging funding environment

Topics include cell based gene therapy, Fabry disease, lentiviral vectors, stem cell engineering, rare disease drug development, regulatory science, and translational medicine.